Validation of the risk stratification score in idiopathic pulmonary fibrosis: study protocol of a prospective, multi-centre, observational, 3-year clinical trial

Abstract Background Idiopathic pulmonary fibrosis (IPF) is characterized by a poor prognosis, with a progressive decline in lung function and considerable variability in the disease’s natural history. Besides lung transplantation (LTx), the only available treatments are anti-fibrosing drugs, which h...

Descripción completa

Guardado en:

Detalles Bibliográficos
Autores principales:	Gian Marco Manzetti, Karishma Hosein, Matthew J. Cecchini, Keith Kwan, Mohamed Abdelrazek, Maurizio Zompatori, Paola Rogliani, Marco Mura
Formato:	article
Lenguaje:	EN
Publicado:	BMC 2021
Materias:	Idiopathic pulmonary fibrosis Prognosis Survival Mortality Lung transplantation Diseases of the respiratory system RC705-779
Acceso en línea:	https://doaj.org/article/d941364ce766476cb72b8b4b0e4d70b8
Etiquetas:	Agregar Etiqueta Sin Etiquetas, Sea el primero en etiquetar este registro!

id	oai:doaj.org-article:d941364ce766476cb72b8b4b0e4d70b8
record_format	dspace
spelling	oai:doaj.org-article:d941364ce766476cb72b8b4b0e4d70b82021-12-05T12:23:28ZValidation of the risk stratification score in idiopathic pulmonary fibrosis: study protocol of a prospective, multi-centre, observational, 3-year clinical trial10.1186/s12890-021-01753-71471-2466https://doaj.org/article/d941364ce766476cb72b8b4b0e4d70b82021-12-01T00:00:00Zhttps://doi.org/10.1186/s12890-021-01753-7https://doaj.org/toc/1471-2466Abstract Background Idiopathic pulmonary fibrosis (IPF) is characterized by a poor prognosis, with a progressive decline in lung function and considerable variability in the disease’s natural history. Besides lung transplantation (LTx), the only available treatments are anti-fibrosing drugs, which have shown to slow down the disease course. Therefore, predicting the prognosis is of pivotal importance to avoid treatment delays, which may be fatal for patients with a high risk of progression. Previous studies showed that a multi-dimensional approach is practical and effective in the development of a reliable prognostic score for IPF. In the RIsk Stratification scorE (RISE), physiological parameters, an objective measure of patient-reported dyspnea and exercise capacity are combined to capture different domains of the complex pathophysiology of IPF. Methods This is an observational, multi-centre, prospective cohort study, designed to reflect common clinical practice in IPF. A development cohort and a validation cohort will be included. Patients newly diagnosed with IPF based on the ATS/ERS criteria and multi-disciplinary discussion will be included in the study. A panel of chest radiologists and lung pathologists will further assess eligibility. At the first visit (time of diagnosis), and every 4-months, MRC dyspnea score, pulmonary function tests (FEV1, FVC and DLCO), and 6-min walking distance will be recorded. Patients will be prospectively followed for 3 years. Comorbidities will be considered. The radiographic extent of fibrosis on HRCT will be recalculated at a 2-year interval. RISE, Gender-Age-Physiology, CPI and Mortality Risk Scoring System will be calculated at 4-month intervals. Longitudinal changes of each variable considered will be assessed. The primary endpoint is 3-year LTx-free survival from the time of diagnosis. Secondary endpoints include several, clinically-relevant information to ensure reproducibility of results across a wide range of disease severity and in concomitance of associated pulmonary hypertension or emphysema. Discussion The objective of this study is to validate RISE as a simple, straightforward, inexpensive and reproducible tool to guide clinical decision making in IPF, and potentially as an endpoint for future clinical trials. Trial registration: U.S National Library of Medicine Clinicaltrials.gov, trial n. NCT02632123 “Validation of the risk stratification score in idiopathic pulmonary fibrosis”. Date of registration: December 16th, 2015.Gian Marco ManzettiKarishma HoseinMatthew J. CecchiniKeith KwanMohamed AbdelrazekMaurizio ZompatoriPaola RoglianiMarco MuraBMCarticleIdiopathic pulmonary fibrosisPrognosisSurvivalMortalityLung transplantationDiseases of the respiratory systemRC705-779ENBMC Pulmonary Medicine, Vol 21, Iss 1, Pp 1-8 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	Idiopathic pulmonary fibrosis Prognosis Survival Mortality Lung transplantation Diseases of the respiratory system RC705-779
spellingShingle	Idiopathic pulmonary fibrosis Prognosis Survival Mortality Lung transplantation Diseases of the respiratory system RC705-779 Gian Marco Manzetti Karishma Hosein Matthew J. Cecchini Keith Kwan Mohamed Abdelrazek Maurizio Zompatori Paola Rogliani Marco Mura Validation of the risk stratification score in idiopathic pulmonary fibrosis: study protocol of a prospective, multi-centre, observational, 3-year clinical trial
description	Abstract Background Idiopathic pulmonary fibrosis (IPF) is characterized by a poor prognosis, with a progressive decline in lung function and considerable variability in the disease’s natural history. Besides lung transplantation (LTx), the only available treatments are anti-fibrosing drugs, which have shown to slow down the disease course. Therefore, predicting the prognosis is of pivotal importance to avoid treatment delays, which may be fatal for patients with a high risk of progression. Previous studies showed that a multi-dimensional approach is practical and effective in the development of a reliable prognostic score for IPF. In the RIsk Stratification scorE (RISE), physiological parameters, an objective measure of patient-reported dyspnea and exercise capacity are combined to capture different domains of the complex pathophysiology of IPF. Methods This is an observational, multi-centre, prospective cohort study, designed to reflect common clinical practice in IPF. A development cohort and a validation cohort will be included. Patients newly diagnosed with IPF based on the ATS/ERS criteria and multi-disciplinary discussion will be included in the study. A panel of chest radiologists and lung pathologists will further assess eligibility. At the first visit (time of diagnosis), and every 4-months, MRC dyspnea score, pulmonary function tests (FEV1, FVC and DLCO), and 6-min walking distance will be recorded. Patients will be prospectively followed for 3 years. Comorbidities will be considered. The radiographic extent of fibrosis on HRCT will be recalculated at a 2-year interval. RISE, Gender-Age-Physiology, CPI and Mortality Risk Scoring System will be calculated at 4-month intervals. Longitudinal changes of each variable considered will be assessed. The primary endpoint is 3-year LTx-free survival from the time of diagnosis. Secondary endpoints include several, clinically-relevant information to ensure reproducibility of results across a wide range of disease severity and in concomitance of associated pulmonary hypertension or emphysema. Discussion The objective of this study is to validate RISE as a simple, straightforward, inexpensive and reproducible tool to guide clinical decision making in IPF, and potentially as an endpoint for future clinical trials. Trial registration: U.S National Library of Medicine Clinicaltrials.gov, trial n. NCT02632123 “Validation of the risk stratification score in idiopathic pulmonary fibrosis”. Date of registration: December 16th, 2015.
format	article
author	Gian Marco Manzetti Karishma Hosein Matthew J. Cecchini Keith Kwan Mohamed Abdelrazek Maurizio Zompatori Paola Rogliani Marco Mura
author_facet	Gian Marco Manzetti Karishma Hosein Matthew J. Cecchini Keith Kwan Mohamed Abdelrazek Maurizio Zompatori Paola Rogliani Marco Mura
author_sort	Gian Marco Manzetti
title	Validation of the risk stratification score in idiopathic pulmonary fibrosis: study protocol of a prospective, multi-centre, observational, 3-year clinical trial
title_short	Validation of the risk stratification score in idiopathic pulmonary fibrosis: study protocol of a prospective, multi-centre, observational, 3-year clinical trial
title_full	Validation of the risk stratification score in idiopathic pulmonary fibrosis: study protocol of a prospective, multi-centre, observational, 3-year clinical trial
title_fullStr	Validation of the risk stratification score in idiopathic pulmonary fibrosis: study protocol of a prospective, multi-centre, observational, 3-year clinical trial
title_full_unstemmed	Validation of the risk stratification score in idiopathic pulmonary fibrosis: study protocol of a prospective, multi-centre, observational, 3-year clinical trial
title_sort	validation of the risk stratification score in idiopathic pulmonary fibrosis: study protocol of a prospective, multi-centre, observational, 3-year clinical trial
publisher	BMC
publishDate	2021
url	https://doaj.org/article/d941364ce766476cb72b8b4b0e4d70b8
work_keys_str_mv	AT gianmarcomanzetti validationoftheriskstratificationscoreinidiopathicpulmonaryfibrosisstudyprotocolofaprospectivemulticentreobservational3yearclinicaltrial AT karishmahosein validationoftheriskstratificationscoreinidiopathicpulmonaryfibrosisstudyprotocolofaprospectivemulticentreobservational3yearclinicaltrial AT matthewjcecchini validationoftheriskstratificationscoreinidiopathicpulmonaryfibrosisstudyprotocolofaprospectivemulticentreobservational3yearclinicaltrial AT keithkwan validationoftheriskstratificationscoreinidiopathicpulmonaryfibrosisstudyprotocolofaprospectivemulticentreobservational3yearclinicaltrial AT mohamedabdelrazek validationoftheriskstratificationscoreinidiopathicpulmonaryfibrosisstudyprotocolofaprospectivemulticentreobservational3yearclinicaltrial AT mauriziozompatori validationoftheriskstratificationscoreinidiopathicpulmonaryfibrosisstudyprotocolofaprospectivemulticentreobservational3yearclinicaltrial AT paolarogliani validationoftheriskstratificationscoreinidiopathicpulmonaryfibrosisstudyprotocolofaprospectivemulticentreobservational3yearclinicaltrial AT marcomura validationoftheriskstratificationscoreinidiopathicpulmonaryfibrosisstudyprotocolofaprospectivemulticentreobservational3yearclinicaltrial
_version_	1718371989334261760

Validation of the risk stratification score in idiopathic pulmonary fibrosis: study protocol of a prospective, multi-centre, observational, 3-year clinical trial

Ejemplares similares